2025-523275-27-00招募中3 期
HELIOS: An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP).
Disc Medicine Inc.4 个研究点 分布在 2 个国家目标入组 14 人开始时间: 2026年2月12日最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 14
- 试验地点
- 4
- 主要终点
- Safety and tolerability of bitopertin, as assessed by the incidence of treatmentemergent adverse events (TEAEs), vital signs, physical examinations, and clinical laboratory parameters.
研究概览
简要总结
To assess the long-term safety and tolerability of bitopertin in participants with EPP (any FECH and ALAS2 genotypes) or X-linked protoporphyria (XLP).
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc bitopertin study and who have completed the randomized treatment phase and End-of- Study visit.
- •Aged ≥12 years upon study consent.
- •Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants.
- •Willing to practice highly effective methods of birth control (both males who have partners of childbearing potential and females of childbearing potential during the study, while taking study drug, and for at least 30 days after the last dose of study drug.
- •Negative urine or serum pregnancy test (females of childbearing potential).
- •Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary).
- •Able to comply with all study procedures.
排除标准
- •Participants who have an ongoing serious adverse event (SAE) from a clinical study that is assessed by the investigator as related to bitopertin.
- •Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation in the study.
- •Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months.
- •Planned treatment with afamelanotide or dersimelagon during the study.
- •Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome P450 (CYP)3A4 enzymes throughout the study.
- •If female, pregnant or breastfeeding.
- •Participation in any other clinical protocol or investigational study, other than Disc bitopertin studies, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day
- •Score of PHQ-8 ≥10 at screening or imminent suicidal risk identified by the C-SSRS as defined as suicidal ideation with intent (Grade 4 or 5) within the last year or any suicidal behavior within the last 5 years.
- •Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study.
结局指标
主要结局
Safety and tolerability of bitopertin, as assessed by the incidence of treatmentemergent adverse events (TEAEs), vital signs, physical examinations, and clinical laboratory parameters.
Safety and tolerability of bitopertin, as assessed by the incidence of treatmentemergent adverse events (TEAEs), vital signs, physical examinations, and clinical laboratory parameters.
次要结局
- 2. Percent change from baseline in whole blood metal-free PPIX concentrations.
- 1. Change from baseline in daylight tolerance, as assessed by total hours spent in sunlight without pain and average time to first prodromal syndrome in sunlight.
- 3. Plasma bitopertin concentrations.
研究者
Disc Medicine Clinical Trials
Scientific
Disc Medicine Inc.
研究点 (4)
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